EP3755795A4 - Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof - Google Patents

Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof Download PDF

Info

Publication number
EP3755795A4
EP3755795A4 EP19755222.7A EP19755222A EP3755795A4 EP 3755795 A4 EP3755795 A4 EP 3755795A4 EP 19755222 A EP19755222 A EP 19755222A EP 3755795 A4 EP3755795 A4 EP 3755795A4
Authority
EP
European Patent Office
Prior art keywords
adeno
restoring
methods
associated virus
gene function
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP19755222.7A
Other languages
German (de)
French (fr)
Other versions
EP3755795A1 (en
Inventor
Saswati Chatterjee
Laura Jane Smith
Jeff Lynn Ellsworth
Hillard RUBIN
Jason Boke WRIGHT
James Anthony Mcswiggen
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
City of Hope
Homology Medicines Inc
Original Assignee
City of Hope
Homology Medicines Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by City of Hope, Homology Medicines Inc filed Critical City of Hope
Publication of EP3755795A1 publication Critical patent/EP3755795A1/en
Publication of EP3755795A4 publication Critical patent/EP3755795A4/en
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10042—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10061—Methods of inactivation or attenuation
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
EP19755222.7A 2018-02-19 2019-02-19 Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof Pending EP3755795A4 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862632300P 2018-02-19 2018-02-19
US201862632919P 2018-02-20 2018-02-20
US201862672385P 2018-05-16 2018-05-16
PCT/US2019/018502 WO2019161365A1 (en) 2018-02-19 2019-02-19 Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof

Publications (2)

Publication Number Publication Date
EP3755795A1 EP3755795A1 (en) 2020-12-30
EP3755795A4 true EP3755795A4 (en) 2022-07-20

Family

ID=67618799

Family Applications (1)

Application Number Title Priority Date Filing Date
EP19755222.7A Pending EP3755795A4 (en) 2018-02-19 2019-02-19 Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof

Country Status (5)

Country Link
US (2) US11306329B2 (en)
EP (1) EP3755795A4 (en)
JP (1) JP7244547B2 (en)
CN (1) CN112041437A (en)
WO (1) WO2019161365A1 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022241041A2 (en) * 2021-05-11 2022-11-17 City Of Hope Adeno-associated virus compositions and methods of use thereof

Family Cites Families (150)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
DE4339922C1 (en) 1993-09-03 1994-10-06 Max Planck Gesellschaft Vector for gene therapy of the liver
US6268212B1 (en) 1993-10-18 2001-07-31 Amgen Inc. Tissue specific transgene expression
DE4407859C1 (en) 1994-03-04 1995-03-02 Max Planck Gesellschaft Vector for liver-specific gene therapy
CA2187626C (en) 1994-04-13 2009-11-03 Michael G. Kaplitt Aav-mediated delivery of dna to cells of the nervous system
AU3718595A (en) 1994-09-16 1996-03-29 Uab Research Foundation, The Transduction of genes into human hematopoietic stem cells using recombinant adeno-associated viral vectors
CA2200835A1 (en) 1994-09-23 1996-03-28 Frederick M. Boyce Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US5650309A (en) 1995-05-16 1997-07-22 The Regents Of The University Of California Viral vectors
US7001764B2 (en) 1995-06-27 2006-02-21 Cell Genesys, Inc. Compositions comprising tissue specific adenoviral vectors
DE19525900C1 (en) 1995-07-15 1996-12-12 Max Planck Gesellschaft Liver-specific adenovirus expression vector
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5780447A (en) 1996-06-14 1998-07-14 St. Jude Children's Research Hospital Recombinant adeno-associated viral vectors
WO1998009524A1 (en) 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
AU4266597A (en) 1996-09-11 1998-04-14 General Hospital Corporation, The Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell
DE19644500C2 (en) 1996-10-25 1998-11-05 Deutsches Krebsforsch AAV DNA with helper virus sequences
FR2755699B1 (en) 1996-11-08 1998-12-18 Rhone Poulenc Rorer Sa NEW CONSTRUCTIONS AND VECTORS FOR TARGETED AND INDUCTIBLE GENE EXPRESSION
FR2756297B1 (en) 1996-11-22 1999-01-08 Centre Nat Rech Scient PROCESS FOR PRODUCING RECOMBINANT VIRUSES
WO1998024479A1 (en) 1996-12-02 1998-06-11 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
JP2001506132A (en) 1996-12-18 2001-05-15 ターゲティッド ジェネティクス コーポレイション Recombinase activatable AAV packaging cassette for use in producing AAV vectors
WO1998028417A1 (en) 1996-12-23 1998-07-02 Gene-Cell Nucleic acid constructs and uses thereof for direct nucleic acid incorporation into cells
US6153436A (en) 1997-01-10 2000-11-28 The Board Of Trustees Of The University Of Arkansas Method of gene delivery using wildtype adeno associated viral (AAV) vectors with insertions
CA2797661C (en) 1997-04-24 2015-06-16 University Of Washington Targeted gene modification by parvoviral vectors
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
GB9715064D0 (en) 1997-07-17 1997-09-24 Ppl Therapeutics Scotland Ltd Protein expression
AU1269699A (en) 1997-10-08 1999-04-27 Advanced Research And Technology Institute, Inc. Chimeric parvovirus-based recombinant vector system that specifically targets the erythroid lineage
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
AU4558799A (en) 1998-06-10 1999-12-30 Advanced Research And Technology Institute, Inc. Methods and compositions for generating recombinant adeno-associated virus vectors
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
US6919209B1 (en) 1998-12-04 2005-07-19 City Of Hope Method of genetically modifying very primitive quiescent human hematopoietic stem cells
JP4693244B2 (en) 1999-03-18 2011-06-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Compositions and methods for helperless production of recombinant adeno-associated virus
US6610906B1 (en) 1999-06-09 2003-08-26 The Regents Of The University Of Michigan Nucleotide sequences for gene regulation and methods of use thereof
ES2284545T3 (en) 1999-11-16 2007-11-16 Genzyme Corporation VECTORS AND TRANSGINS WITH REGULATING ELEMENTS FOR THE ADMINISTRATION OF LIVER GENES.
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
ES2256265T3 (en) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill DUVICATED PARVOVIRUS VECTORS.
US7351813B2 (en) 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
WO2002066611A2 (en) 2001-02-16 2002-08-29 The Board Of Trustees Of The Leland Stanford Junior University Minimal plasmid vectors that provide for persistent and high level gene expression and methods for using the same
DE10120265A1 (en) 2001-04-25 2002-11-14 Deutsches Krebsforsch AAV helper plasmids for helper virus-free packaging and pseudotyping of AAV vectors
DE60233047D1 (en) 2001-05-14 2009-09-03 Gbp Ip Llc LENTIVIRAL VECTORS ENCODING FLAMMABLE FACTORS FOR GENETHERAPY
AU2002305778A1 (en) 2001-05-31 2002-12-09 The Rockefeller University Method for generating replication defective viral vectors that are helper free
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
US20030129203A1 (en) 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
JP2005526001A (en) 2001-10-04 2005-09-02 アグイラルーコルドバ，カリオス，エスツアルド Chimeric virus vector for gene therapy
CA2361462A1 (en) 2001-11-07 2003-05-08 Katherine A. High Induction of tolerance to a therapeutic polypeptide
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
PT1453547T (en) 2001-12-17 2016-12-28 Univ Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
JP4769417B2 (en) 2001-12-17 2011-09-07 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Adeno-associated virus (AAV) serotype 9 sequences, vectors containing the same and uses thereof
CA2369985A1 (en) 2002-01-18 2003-07-18 Duke University Generation of recombinant adeno-associated viral vectors by a complete adenovirus-mediated approach
JP3943048B2 (en) 2002-04-29 2007-07-11 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Method for direct rescue and amplification of integrated virus from cellular DNA of tissue
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US7220577B2 (en) 2002-08-28 2007-05-22 University Of Florida Research Foundation, Inc. Modified AAV
US7091029B2 (en) 2002-09-23 2006-08-15 Applied Genetics Technologies Corporation High titer recombinant AAV production
US20070202587A1 (en) 2002-09-23 2007-08-30 Applied Genetic Technologies Corporation Recombinant AAV production in mammalian cells
DK3235827T3 (en) 2003-06-19 2021-04-19 Genzyme Corp AAV VIRIONS WITH REDUCED IMMUNE ACTIVITY AND ITS APPLICATIONS
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
ES2648241T3 (en) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Adeno-associated virus clades (AAV), sequences, vectors containing the same, and uses thereof
US7482156B2 (en) 2003-10-15 2009-01-27 Cell Genesys, Inc. Hepatocellular carcinoma specific promoter and uses thereof
EP1732383A4 (en) 2004-04-06 2007-05-02 Cedars Sinai Medical Center Prevention and treatment of vascular disease with recombinant adeno-associated virus vectors encoding apolipoprotein a-i and apolipoprotein a-i milano
WO2006065273A2 (en) 2004-05-24 2006-06-22 The Regents Of The University Of California New human parvovirus
WO2006036502A2 (en) 2004-09-22 2006-04-06 St. Jude Children's Research Hospital Improved expression of factor ix in gene therapy vectors
TWI293307B (en) 2004-09-30 2008-02-11 Ind Tech Res Inst A liver-specific chimeric regulatory sequence and use thereof
KR20070111542A (en) 2005-03-09 2007-11-21 보드 오브 리전츠, 더 유니버시티 오브 텍사스 시스템 Novel htmc promoter and vectors for the tumor-selective and high-efficient expression of cancer therapeutic genes
ES2525067T3 (en) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Method of increasing the function of an AAV vector
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
WO2007019646A1 (en) 2005-08-19 2007-02-22 University Of Technology, Sydney Liver-directed gene therapy
AU2006304997B2 (en) 2005-10-20 2012-03-01 Uniqure Ip B.V. Improved AAV vectors produced in insect cells
WO2007084773A2 (en) 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
EP1979485A2 (en) 2006-01-31 2008-10-15 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
WO2007127428A2 (en) 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
PL2191001T3 (en) 2007-04-09 2017-01-31 University Of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
EP1985631A1 (en) * 2007-04-20 2008-10-29 LFB Biotechnologies Demannosylated recombinant factor VIII for the treatment of patients with hemophiila A
US10696983B2 (en) 2007-05-30 2020-06-30 The Trustees Of The University Of Pennsylvania Method for transducing cells with primary cilia
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US20090215879A1 (en) 2008-02-26 2009-08-27 University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
EP4154903A1 (en) 2008-04-22 2023-03-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
EP2315833B8 (en) 2008-05-20 2015-05-27 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
PL2425000T3 (en) 2009-04-30 2019-08-30 The Trustees Of The University Of Pennsylvania Compositions for targeting conducting airway cells comprising adeno-associated virus constructs
EP2435575A2 (en) 2009-05-28 2012-04-04 Deutsches Krebsforschungszentrum Modified aav capsid polypeptides
HUE028341T2 (en) 2009-06-16 2016-12-28 Genzyme Corp Improved methods for purification of recombinant aav vectors
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP2287323A1 (en) 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
WO2011041502A1 (en) 2009-10-01 2011-04-07 The Trustees Of The University Of Pennsylvania Aav vectors expressing sec1o for treating kidney damage
WO2011088081A1 (en) 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
CN105838737A (en) 2010-01-28 2016-08-10 费城儿童医院 A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
US9546112B2 (en) 2010-03-22 2017-01-17 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
CA2793633A1 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
US9839696B2 (en) * 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
US20120014932A1 (en) 2010-07-14 2012-01-19 Golden William J Methods for treating disorders of amino acid metabolism
US9409953B2 (en) 2011-02-10 2016-08-09 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
US10392632B2 (en) 2011-02-14 2019-08-27 The Children's Hospital Of Philadelphia AAV8 vector with enhanced functional activity and methods of use thereof
BR112013020734A2 (en) 2011-02-17 2017-06-13 Univ Pennsylvania compositions and methods for altering tissue specificity and enhancing aav9-mediated gene transfer
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
WO2012122025A2 (en) 2011-03-04 2012-09-13 Intrexon Corporation Vectors conditionally expressing protein
PL3254703T3 (en) 2011-04-22 2020-10-05 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
EP2771457B1 (en) 2011-10-27 2017-11-22 Sangamo Therapeutics, Inc. Methods and compositions for modification of the hprt locus
US10640785B2 (en) 2011-11-22 2020-05-05 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
US20140359799A1 (en) 2011-12-23 2014-12-04 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
CA2864879C (en) 2012-02-17 2021-07-20 The Children's Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues
PT2839014T (en) 2012-04-18 2021-03-19 Childrens Hospital Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
BR112015000161A2 (en) 2012-07-06 2017-06-27 Univ Iowa Res Found modified adeno-associated virus vector compositions
CN104797593B (en) 2012-09-28 2020-05-08 北卡罗来纳-查佩尔山大学 AAV vectors targeting oligodendrocytes
AU2013336601B2 (en) 2012-10-26 2018-01-25 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
US9255250B2 (en) 2012-12-05 2016-02-09 Sangamo Bioscience, Inc. Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene
WO2014089541A2 (en) 2012-12-07 2014-06-12 Haplomics, Inc. Factor viii mutation repair and tolerance induction
US9938541B2 (en) 2012-12-25 2018-04-10 Takara Bio Inc. AAV variant
WO2014109728A1 (en) 2013-01-11 2014-07-17 The Scripps Research Institute Methods and compositions for enchancing transduction efficiency of retroviral vectors
WO2014124282A1 (en) 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
AU2014244167A1 (en) 2013-03-13 2015-10-08 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
US20140271550A1 (en) 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
JP6591956B2 (en) 2013-03-15 2019-10-16 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Compositions and methods for treating MPS1
CA2904156C (en) 2013-03-15 2023-01-10 The Children's Hospital Of Philadelphia Vectors comprising stuffer/filler polynucleotide sequences and methods of use
CA2917018A1 (en) 2013-05-21 2014-12-04 University Of Florida Research Foundation, Inc. Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
CA2907799A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
EP3019620A4 (en) 2013-07-12 2017-01-18 The Children's Hospital of Philadelphia Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies
WO2015013313A2 (en) 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2015038958A1 (en) 2013-09-13 2015-03-19 California Institute Of Technology Selective recovery
CN105899665B (en) 2013-10-17 2019-10-22 桑格摩生物科学股份有限公司 The delivering method and composition being transformed for nuclease-mediated genome project
AU2015231231B2 (en) 2014-03-21 2021-09-02 The Board Of Trustees Of The Leland Stanford Junior University Genome editing without nucleases
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
ES2856090T3 (en) 2014-09-24 2021-09-27 Hope City Adeno-associated virus vector variants for high-efficiency genomic editing and their methods
EP3233131A1 (en) 2014-12-16 2017-10-25 Board of Regents of the University of Nebraska Gene therapy for juvenile batten disease
JP6956635B2 (en) 2014-12-17 2021-11-02 フンダシオン パラ ラ インベスティガシオン メディカ アプリカダ Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson's disease and other conditions
IL252917B2 (en) 2014-12-17 2023-10-01 Fundacion Para La Investig Medica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson disease
US10308705B2 (en) 2015-02-06 2019-06-04 The University Of North Carolina At Chapel Hill Optimized human clotting factor VIII gene expression cassettes and their use
EP3270944B1 (en) 2015-03-17 2019-10-23 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix
CN104659074B (en) 2015-03-17 2019-04-09 京东方科技集团股份有限公司 A kind of oled substrate, its manufacturing method, panel and display device
CN115074366A (en) 2015-04-16 2022-09-20 埃默里大学 Recombinant promoter and vector for protein expression in liver and application thereof
BR112018008519A2 (en) 2015-10-28 2018-11-06 Sangamo Therapeutics Inc liver-specific constructs, factor viii expression cassettes and methods of use thereof
US11389546B2 (en) 2015-12-09 2022-07-19 Modernatx, Inc. Heterologous UTR sequences for enhanced mRNA expression
KR102604096B1 (en) 2016-12-30 2023-11-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Gene therapy to treat Wilson's disease
AU2018207259B2 (en) 2017-01-10 2023-11-09 Children's Medical Research Institute Polynucleotides and vectors for the expression of transgenes
US20190010517A1 (en) 2017-07-06 2019-01-10 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for facilitating homologous recombination

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
A. BARZEL ET AL: "Promoterless gene targeting without nucleases ameliorates haemophilia B in mice", NATURE, vol. 517, no. 7534, 29 October 2014 (2014-10-29), pages 360 - 364, XP055174017, ISSN: 0028-0836, DOI: 10.1038/nature13864 *
CHUL-YONG PARK ET AL: "Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9", CELL STEM CELL, vol. 17, no. 2, 1 August 2015 (2015-08-01), AMSTERDAM, NL, pages 213 - 220, XP055382943, ISSN: 1934-5909, DOI: 10.1016/j.stem.2015.07.001 *
IRAM F KHAN ET AL: "Engineering of Human Pluripotent Stem Cells by AAV-mediated Gene Targeting", MOLECULAR THERAPY, vol. 18, no. 6, 1 June 2010 (2010-06-01), US, pages 1192 - 1199, XP055489061, ISSN: 1525-0016, DOI: 10.1038/mt.2010.55 *
LAURA J. SMITH ET AL: "Stem cell-derived clade F AAVs mediate high-efficiency homologous recombination-based genome editing", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 115, no. 31, 17 July 2018 (2018-07-17), pages E7379 - E7388, XP055609078, ISSN: 0027-8424, DOI: 10.1073/pnas.1802343115 *
MCINTOSH JENNY ET AL: "Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant", BLOOD, AMERICAN SOCIETY OF HEMATOLOGY, US, vol. 121, no. 17, 25 April 2013 (2013-04-25), pages 3335 - 3344, XP086510295, ISSN: 0006-4971, [retrieved on 20201119], DOI: 10.1182/BLOOD-2012-10-462200 *
See also references of WO2019161365A1 *
YONG WU ET AL: "In situ genetic correction of F8 intron 22 inversion in hemophilia A patient-specific iPSCs", SCIENTIFIC REPORTS, vol. 6, no. 1, 8 January 2016 (2016-01-08), XP055382937, DOI: 10.1038/srep18865 *

Also Published As

Publication number Publication date
US11306329B2 (en) 2022-04-19
US11891619B2 (en) 2024-02-06
US20220333138A1 (en) 2022-10-20
US20190276856A1 (en) 2019-09-12
JP2021513871A (en) 2021-06-03
JP7244547B2 (en) 2023-03-22
CN112041437A (en) 2020-12-04
WO2019161365A1 (en) 2019-08-22
EP3755795A1 (en) 2020-12-30

Similar Documents

Publication Publication Date Title
EP3684423A4 (en) Adeno-associated virus variant capsids and methods of use thereof
EP3445773A4 (en) Adeno-associated virus variant capsids and methods of use thereof
EP3856913A4 (en) Adeno-associated virus compositions for targeted gene therapy
EP3676373A4 (en) Adeno-associated virus capsid variants and methods of use thereof
EP3790596A4 (en) Oligonucleotide compositions and methods of use thereof
EP3823673A4 (en) Anti-cd112r compositions and methods
IL287214A (en) Adeno-associated virus vector formulations and methods
EP3980436A4 (en) Nucleic acid-polypeptide compositions and uses thereof
IL273877A (en) Adeno-associated virus compositions for restoring hbb gene function and methods of use thereof
EP3735224A4 (en) Compositions comprising co-selected microbiota and methods for use thereof
EP3759218A4 (en) Methods and compositions for antibody-evading virus vectors
EP3746113A4 (en) Adeno-associated virus compositions for pah gene transfer and methods of use thereof
EP3807297A4 (en) Synthetic liver-tropic adeno-associated virus capsids and uses thereof
EP3575398A4 (en) Mutant of adeno-associated virus (aav) capsid protein
EP3790861A4 (en) Senolytic compositions and uses thereof
EP3829307A4 (en) Bismuth-thiol compositions and methods of use
IL276368A (en) Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
EP3500278A4 (en) Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus
EP3793563A4 (en) Compositions comprising bisfluoroalkyl-1,4-benzodiazepinone compounds and methods of use thereof
EP3768315A4 (en) Fc variant compositions and methods of use thereof
EP3844500A4 (en) Rp182 compositions and methods
EP3765075A4 (en) Hepatitis c virus peptide compositions and methods of use thereof
EP3790552A4 (en) Combination compositions comprising bisfluoroalkyl-1,4- benzodiazepinone compounds and methods of use thereof
EP3840769A4 (en) Cyclosporine compositions and methods of use
EP3500279A4 (en) Methods and compositions for treating canine conditions using recombinant self-complementary adeno-associated virus

Legal Events

Date Code Title Description
STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE

PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE

17P Request for examination filed

Effective date: 20200918

AK Designated contracting states

Kind code of ref document: A1

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

AX Request for extension of the european patent

Extension state: BA ME

DAV Request for validation of the european patent (deleted)
DAX Request for extension of the european patent (deleted)
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40043675

Country of ref document: HK

RIC1 Information provided on ipc code assigned before grant

Ipc: A61K 48/00 20060101ALI20220314BHEP

Ipc: C12N 15/09 20060101ALI20220314BHEP

Ipc: C12N 15/00 20060101AFI20220314BHEP

A4 Supplementary search report drawn up and despatched

Effective date: 20220622

RIC1 Information provided on ipc code assigned before grant

Ipc: A61K 48/00 20060101ALI20220615BHEP

Ipc: C12N 15/09 20060101ALI20220615BHEP

Ipc: C12N 15/00 20060101AFI20220615BHEP

P01 Opt-out of the competence of the unified patent court (upc) registered

Effective date: 20230414